Behind the Scenes: A thread of randomized trials I have helped lead and publish. Each one has a story. Most have been a struggle.
This is for young physicians trying to start a career as a clinical investigator or trialist.
#MedTwitter #MedStudentTwitter #myelomaVR #mmsm
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1) ECOG Thalidomide Dex vs Dex for myeloma. My first RCT. I had previously led small Thal trials at Mayo. But leading this pivotal RCT required luck. I had proposed a small Ph II, so they let me be PI. But @theNCI mandated a change to Ph III RCT design. ascopubs.org/doi/10.1200/JC…
2) Celgene Thal Dex vs Dex placebo RCT. Given success of ECOG trial, which led to thalidomide approval by FDA, I was asked by Celgene to lead their company trial: a confirmatory trial for regulatory approval. Company trials are easier to run. ascopubs.org/doi/10.1200/JC…
3) ECOG high dose vs low dose dexamethasone: A patient, the late Michael Katz proposed this trial. Glad we listened. Results led to a dramatic reduction in myeloma deaths & morbidity worldwide. Also gave Rd backbone used in most regimens. @TheLancetOncol thelancet.com/journals/lanon…
4) ECOG MPT vs MPR: Slow accrual. So by the time the results of this trial came, melphalan based regimens were out of favor. Shows how long delays in opening, and slow accrual that affect many RCTs in the US. @akeithstewart the trial PI did all the work. ashpublications.org/blood/article/…
5) Mayo Clinic Thalidomide Zoledronic Acid vs Zoledronic acid for smoldering myeloma. Managed to get @NIH R01 grant to fund an RCT. Not just lab correlatives, but actual trial costs! These grants are scarce now. Precursor to Lenalidomide smoldering RCT. nature.com/articles/leu20…
6) Takeda Ixazomib vs Placebo Maintenance RCT. Great lessons from interactions with FDA and the sponsor on trial design and endpoints for maintenance. Novel design with early read out of PFS, but continue trial for OS endpoint. @TheLancet thelancet.com/journals/lance…
7) ECOG Lenalidomide versus Observation for smoldering myeloma. I’ve written a long thread about the 15 year saga. This trial together with the RCT by @mvmateos changed paradigm for high risk smoldering myeloma. Great working with trial PI @SagarLonialMD ascopubs.org/doi/10.1200/JC…
8) ECOG ENDURANCE VRd vs KRd for myeloma. 10 year effort. Was struggle to open. But accrued fast. Results show why RCTs are important. It’s also 2 RCTs in one! 2nd one: Len 2 years vs Len till progression is ongoing. Great working with trial PI @myelomaMD thelancet.com/journals/lanon…
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